C12N2799/022

Tumor-targeting synthetic adenoviruses and uses thereof

Synthetic adenoviruses with liver detargeting mutations and expressing an adenovirus type 34 (Ad34) fiber protein, or a chimeric fiber protein with an Ad34 knob domain, are described. The synthetic adenoviruses traffic to sites of tumors. Use of the synthetic adenoviruses for delivering diagnostic or therapeutic transgenes to tumors are also described.

NERVE REGENERATION
20220241433 · 2022-08-04 ·

The present application describes a method of regenerating nerve, which steps include generating a recombinant viral or plasmid vector comprising a DNA sequence encoding a member of a transforming growth factor superfamily of proteins operatively linked to a promoter; transfecting in vitro a population of cultured cells with the recombinant vector, resulting in a population of the cultured cells; and transplanting the transfected cells to an area near an injured nerve, such that expression of the DNA sequence within the area near the injured nerve causes regeneration of the nerve.

CCR3 MODULATION IN THE TREATMENT OF AGING-ASSOCIATED IMPAIRMENTS, AND COMPOSITIONS FOR PRACTICING THE SAME

Methods of treating an adult mammal for an aging-associated impairment are provided. Aspects of the methods include modulating CCR3, e.g., by modulating eotaxin-1/CCR3 interaction, in the mammal in a manner sufficient to treat the mammal for the aging-associated impairment. A variety of aging-associated impairments may be treated by practice of the methods, which impairments include cognitive impairments.

GENE THERAPY FOR OCULAR DISORDERS
20210330816 · 2021-10-28 ·

Compositions and methods are provided for treating ocular disorders in a subject are provided. In one aspect, an adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding CNGA3. In another aspect, an adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding CNGB3. In another aspect, an adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding REP-1. In desired embodiments, the subject is human, cat, dog, sheep, or non-human primate.

Methods of reprogramming cells

The present invention provides methods of reprogramming cells, for example, directly reprogramming a somatic cell of a first cell type into a somatic cell of a second cell type, are described herein. In particular, the present invention generally relates to methods for reprogramming a cell of an endoderm origin to a cell having pancreatic β-cell characteristics. The present invention also relates to an isolated population comprising reprogrammed cells, compositions and their use in the treatment of diabetes mellitus. In particular, the present invention relates to reprogramming a cell of an endoderm origin to a cell having pancreatic β-cell characteristics by increasing the protein expression of at least one transcription factor selected from Pdx1, Ngn3 or MafA in the cell of endoderm origin to reprogram the cell of an endoderm cell to a cell which exhibits at least one or at least two characteristics of an endogenous pancreatic β-cell.

TRANSCRIPTION FACTOR-BASED GENERATION OF PACEMAKER CELLS AND METHODS OF USING SAME
20210260158 · 2021-08-26 · ·

Several embodiments disclosed herein relate generally to methods and compositions for the generation of biological pacemakers. In some embodiments, the methods comprise contacting non-pacemaker cells with one or more transcription factors (in vivo or in vitro) and inducing pacemaker functionality in the cells.

Process for recovering viral products using functionalised chromatography media
11103853 · 2021-08-31 · ·

The present invention provides a process for recovering a viral product from a composition comprising said product and product-related impurities, which process comprises contacting the composition with a functionalised chromatography medium comprising one or more polymer nanofibres, wherein the viral product comprises a plurality of viruses, virus particles/virions, viral cores, membrane-stripped viruses, viral cores with outer membrane(s) removed and/or capsids removed, or proviruses, each of which contains one or more polynucleotides, and wherein the product-related impurities comprise a plurality of viruses, virus particles/virions, virus-like particles, viral cores, membrane-stripped viruses, viral cores with outer membrane(s) removed and/or capsids removed or proviruses, each of which is substantially devoid of polynucleotides.

Synergistic Genome-Nonintegrating Reprogramming by Micrornas and Transcription Factors
20210238556 · 2021-08-05 ·

Disclosed herein are methods of generating induced pluripotent stem cells. The method involves providing a quantity of somatic or non-embryonic cells, contacting the contacting the somatic or non-embryonic cells with a quantity of one or more programming factors and one or more RNA molecules, and culturing the somatic or non-embryonic cells for a period of time sufficient to generate at least one induced pluripotent stem cell. Various reprogramming factors and RNA molecules for use in the methods are disclosed herein. Also disclosed are cell lines and pharmaceutical compositions generated by use of the methods.

Synergistic genome-nonintegrating reprogramming by microRNAs and transcription factors

Disclosed herein are methods of generating induced pluripotent stem cells. The method involves providing a quantity of somatic or non-embryonic cells, contacting the contacting the somatic or non-embryonic cells with a quantity of one or more reprogramming factors and one or more RNA molecules, and culturing the somatic or non-embryonic cells for a period of time sufficient to generate at least one induced pluripotent stem cell. Various reprogramming factors and RNA molecules for use in the methods are disclosed herein. Also disclosed are cell lines and pharmaceutical compositions generated by use of the methods.

Transcription factor-based generation of pacemaker cells and methods of using same

Several embodiments disclosed herein relate generally to methods and compositions for the generation of biological pacemakers. In some embodiments, the methods comprise contacting non-pacemaker cells with one or more transcription factors (in vivo or in vitro) and inducing pacemaker functionality in the cells.